Advancing Hope

Through Science

Since the early days of our journey with Ryan's therapy, the research and science behind mesenchymal stem cell (MSC) therapy have advanced significantly. We are more confident than ever that this innovative approach deserves serious consideration as a potential treatment for various conditions, including Duchenne Muscular Dystrophy (DMD). Below, we present the latest statistics and evidence supporting our position.

Global Clinical Trials Using MSC Therapy

As of May 18, 2021:

Total stem cell therapy clinical trials worldwide: 6,205

Total MSC therapy-related clinical trials worldwide: 1,240

Promising Developments

Recent advancements in MSC therapy for DMD include:

1. Safety Profile: Multiple clinical studies have demonstrated the safety of MSC therapy across various indications.

2. Preliminary Efficacy: Clinical studies using umbilical cord-derived MSCs (UC-MSCs) have shown promising signs of efficacy, including improvements in upper and lower body muscle strength.

3. Long-term Benefits: As part of an FDA approved Investigational New Drug Compassionate Use Exemption - A case study of a 35-year-old DMD patient receiving recurrent UC-MSC treatments over a decade showed remarkably stable pulmonary function.

Additional Reference

4. Pediatric Applications: As part of an FDA approved Investigational New Drug Compassionate Use Exemption - A 4-year-old DMD patient showed improvements in limb coordination and decreased Creatine Kinase enzyme levels one year after receiving intramuscular UC-MSC injections.

Regulatory Landscape

The regulatory environment for mesenchymal stem cell (MSC) therapy in the United States is evolving, potentially expanding access to regenerative therapies. Several key developments are shaping this landscape:

Become An Advocate

Signed into law in 2018, this Act allows patients with life-threatening conditions to access certain unapproved treatments, including some stem cell therapies, outside of clinical trials. Key points include:
- Eligibility for patients who have exhausted approved treatment options and cannot participate in clinical trials
- Requires only Phase 1 safety trials to be completed
- Bypasses certain FDA approval processes for experimental treatments
Passed in 2016, this Act aims to accelerate medical product development and bring innovations to patients faster. It includes provisions for:
- Expedited review processes for regenerative medicine advanced therapies (RMAT)
- Increased funding for research in regenerative medicine
Introduced in 2017, this framework aims to:
- Provide clarity on which regenerative medicine products are subject to FDA premarket review
- Streamline and expedite the approval process for certain regenerative medicine therapies
Passed in 2017, this state-level bill:
- Allows patients with chronic and terminal illnesses to access stem cell treatments that have completed FDA Phase 1 safety trials
- Requires treatments to be administered by physicians at medical facilities
- Mandates informed consent from patients
While not regulatory in nature, these guidelines influence policy and practice:
- Provide ethical and scientific standards for stem cell research and clinical translation
- Updated periodically to address emerging issues in the field
Despite these advancements, challenges remain:
- Balancing safety concerns with the need for innovative treatments
- Harmonizing regulations across different countries
- Addressing the rise of unproven stem cell treatments and medical tourism
The regulatory landscape continues to evolve, with potential developments including:
- Further refinement of expedited approval pathways for regenerative medicine
- Increased international collaboration on regulatory standards
- Development of more specific guidelines for MSC-based therapies
As the field of MSC therapy advances, it's crucial for patients, researchers, and healthcare providers to stay informed about these regulatory developments. They play a significant role in shaping the accessibility and safety of these promising treatments.